Presentation (CTI)

OBJECTIVES: To examine the evolving role of real-world data (RWD) in supporting market access activities and launch strategies for health technologies and pharmaceutical products. This review synthesizes recent regulatory guidance and industry perspectives on integrating RWD into planning processes and value communication strategies.
METHODS: Review of recent publications and regulatory guidance including FDA's 2023 RWD guidance for drug development, EMA's guidance on RWD generation, and industry position papers. Focus on literature published 2022-2025 addressing RWD applications in health technology assessment (HTA).
RESULTS: After reviewing positioning from a range of HTA bodies globally, recent guidance emphasizes the increasing utility of RWD in characterizing patient populations, treatment patterns, and real-world effectiveness to support development of value propositions throughout the product lifecycle and augment HTA evidence packages. Emerging technologies including AI-assisted data linkage and machine learning enable construction of virtual control arms and comprehensive patient characterization, particularly valuable in challenging market access scenarios (e.g., rare disease) therapies, where traditional randomized comparators are unavailable or evidence generation is time-intensive. There is increasing expectation and requirement from national health systems for RWD‑informed launch dossiers alongside traditional clinical data, although there is some nuance (e.g., compulsory only for specific drug classes, therapeutic areas, and high-cost drugs). RWD strengthens payer negotiations by demonstrating post-launch effectiveness and treatment durability, while robust health outcome data (e.g., electronic health records, patient registries) complement clinical trial evidence.
CONCLUSIONS: Integration of well-designed and timely RWD studies into HEOR planning is increasingly important for achieving favorable reimbursement outcomes and faster access pathways, especially in rare diseases. Organizations will benefit from developing integrated evidence plans encompassing transparent and robust RWD within their product development and launch strategy, aligned to the broader HTA and regulatory requirements. Future HEOR strategy should regard RWD generation as foundational infrastructure to support product value demonstration and improve patient access.